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Research News from CHOP

Research News from CHOP

Researcher viewing a sample in a vial
The latest news from CHOP about our research.
News

Researchers at Children’s Hospital of Philadelphia Show Gene Therapy Can Improve Quality of Life for Companion Dogs with Hemophilia

In a study published in the journal Molecular Therapy Methods and Clinical Development, researchers at Children’s Hospital of Philadelphia (CHOP), in collaboration with researchers at the School of Veterinary Medicine at the University of Pennsylvania, found that adeno-associated viral (AAV) gene therapy has the potential to be a life-changing treatment for companion dogs –pets – with severe hemophilia, an inherited bleeding disorder caused by deficiency of clotting factor.

News

Children’s Hospital of Philadelphia Researchers Discover that Acute Myeloid Leukemia Can Permanently Reprogram Stem Cells Through Inflammation

New research from Children’s Hospital of Philadelphia (CHOP) uncovered a previously unknown side effect of Acute Myeloid leukemia (AML), the second most common blood cancer in children, that leaves a long-lasting imprint on healthy blood-forming stem cells, influencing immune responses long after the disease has entered remission. The results were published recently in the journal Cancer Letters.

Stage at Cardiology 2025
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The Cardiac Center at Children’s Hospital of Philadelphia Hosts the 28th Annual Update on Pediatric and Congenital Cardiovascular Disease

Cardiology 2025, the 28th Annual Update on Pediatric and Congenital Cardiovascular Disease, was held this year from February 19 to 23 at Disney’s Yacht & Beach Club Report in Lake Buena Vista, Florida. The conference, hosted by Children’s Hospital of Philadelphia (CHOP), brought together a dynamic and diverse community of pediatric and congenital cardiovascular professionals and advocates, all united by this year’s theme: Hope, Heal and Learn.

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Pediatric Hospital Study Links Structural and Social Drivers of Health to Increased Recurrent Violence in Children with Firearm Injuries

Researchers in the Center for Violence Prevention (CVP) at Children’s Hospital of Philadelphia (CHOP) collaborated with a team led by Lurie Children’s Hospital in Chicago that found children living in areas with the lowest Child Opportunity Index (COI), a measure that assesses factors like education, socioeconomic resources and access to health environments, were more than twice as likely to experience another violent injury within a year of an initial firearm related emergency department (ED) visit. The retrospective study was published recently in the journal JAMA Pediatrics.

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Children’s Hospital of Philadelphia Preclinical Study Demonstrates Improved Gene Therapy Approach to Treating Metachromatic Leukodystrophy

In a preclinical study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased effectiveness and safety for the treatment for metachromatic leukodystrophy (MLD), a rare disease in young children characterized by the deficient activity of a critical enzyme. Without effective treatment, MLD leads to rapidly declining neurological skills in young children and is potentially fatal. The promising findings were published in the journal Molecular Therapy Nucleic Acids.

Go Go Gadget on stage at Cheers for CHOP
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Children’s Hospital of Philadelphia Raises More Than $745,000 for Rare Disease Research at Cheers for CHOP

On Saturday, February 22, 2025, Children's Hospital of Philadelphia (CHOP) hosted Cheers for CHOP, presented by Calvin Schmidt and family, at The Fillmore. The event benefitted rare disease research at CHOP, where rare cases are not so uncommon. Every dollar of the $745,000 (and counting!) raised will help our teams continue their work of translating rare disease research into more precise and personalized care for patients with some of the most complex medical cases.

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Children’s Hospital of Philadelphia Researchers Find New Protocol to Help Stabilize Youth Boarding in Emergency Rooms

Children’s Hospital of Philadelphia (CHOP) researchers successfully reduced the number of youths needing to be admitted to psychiatric facilities through a stabilization protocol designed to address psychiatric boarding, the practice of maintaining patients with psychiatric emergencies in the Emergency Department (ED) or other temporary settings due to a lack of available inpatient psychiatric beds. The report was published recently in the journal Pediatrics.

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